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Advancing Medicines for Neglected and Rare Diseases Through Innovative Business Models
OTC: KBIO www.kalobios.com
| Corporate Overview 1
Forward-Looking Statements
| Corporate Overview 2
This presentation contains forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.Forward-looking statements reflect management’s current knowledge, assumptions, judgment and expectations regarding future performance or events.Although management believes that the expectations reflected in such statements are reasonable, they give no assurance that such expectations will prove to becorrect, and actual results could differ materially from the forward-looking statements.
Words such as “will,” “expect,” “intend,” “plan,” “predict,” “potential,” “possible,” and similar expressions identify forward-looking statements, including, withoutlimitation, statements related to the scope, progress, expansion, and costs of developing and commercializing our product candidates, anticipated regulatoryincentives for product candidates, and anticipated expenses related to development activities, clinical trials and the development and potential commercializationof product candidates.
Forward-looking statements are subject to risks and uncertainties including, but not limited to, the Company’s ability to execute its revised strategy and businessplan; the ability of the Company to list its common stock on a national securities exchange; the Company’s access to limited cash reserves and its ability toobtain additional capital, including the additional capital which will be necessary to complete the clinical trials that the Company has initiated or plans to initiate;the potential timing and outcomes of clinical studies of benznidazole, lenzilumab, ifabotuzumab or any other product candidates; the commercial viability of theCompany’s proposed drug pricing program; the ability of the Company to timely source adequate supply of its development products from third-partymanufacturers on whom the Company depends; the potential, if any, for future development of any of its present or future products; the Company's ability tosuccessfully progress, partner or complete further development of its programs; the ability of the Company to identify and develop additional products; theuncertainties inherent in clinical testing; the timing, cost and uncertainty of obtaining regulatory approvals and reaching other development milestones; theCompany’s ability to apply or receive Priority Review Vouchers; the Company's ability to protect the Company's intellectual property; competition; and changes inthe regulatory landscape or the imposition of regulations that affect the Company's products; and the various risks described in the "Risk Factors" and elsewherein the Company's periodic and other filings with the Securities and Exchange Commission.
You are cautioned not to place undue reliance on any forward-looking statements, which speak only as of the date of this release. The company has noobligation, and expressly disclaims any obligation to update, revise or correct any of the forward-looking statements, whether as a result of new information,future events or otherwise.
Turnaround focused on high-value neglected and rare diseases, including pediatric
Pipeline with multiple potential near- and longer-term catalysts
• Benznidazole: 505(b)(2) development pathway for Chagas disease; possible PRV
• Lenzilumab: recombinant mAb; possible PRV
• Ifabotuzumab: recombinant mAb; pediatric and orphan blood and solid cancers; possible PRV; ready for partnering
Experienced, focused new management with track record of efficient execution, commitment to leadership in responsible, transparent pricing
Compelling Investment Opportunity
| Corporate Overview 3
LeadershipCameron Durrant, MD, MBAChairman and CEOo Senior pharmaceutical and biotech exec, turnaround
specialisto Senior exec roles at Pharmacia/Pfizer, J+J in US,
Merck, GSK in Europe; experience as Exec Chairman, CEO and CFO; CEO roles at three specialty pharma groups
o Expertise in anti-infectives, pediatrics, oncology
Morgan LamChief Scientific Officero Extensive industry experience in clinical researcho Head of Clinical Operations and Development
KaloBioso Executive Director, Medical Affairs, Geron
Dave Tousley, MBA, CPAInterim Chief Financial Officero More than 35 years experience in biotech, spec
pharma, big pharmao Senior exec roles, President, COO, CFOo Pasteur, Merieux, Connaught, AVAX, airPharma,
PediaMed, DARA Biosciences
Tariq Arshad, MD, MBAConsultant focused on medical affairso Extensive industry experience in clinical developmento Experienced in orphan, pediatrics, oncology, pediatric oncology, immunologyo Pharmacia/Pfizer, Genentech, Xoma
Niv Caviar, MBAConsultant focused on corporate/business developmento Senior functional roles in marketing, business development, strategic planningo Senior exec roles, CEO, EVP-CBO, CFO, VP Bus Devo La Jolla Pharma, Allergan, Suneva, SpineOvations, Affymetrix, Accenture
Christopher BoweConsultant focused on corporate affairso Deep experience advising CEOs on articulating, executing strategy through
corporate affairso Former Strategic Affairs advisor at Schering-Plougho Industry thought leader, prior award-winning writer Financial Times
Steve Pal, MBAConsultant focused on commercialo Global pharma and consumer healthcare product commercialisationo Former Corporate VP Global Strategic Marketing, Health Outcomes, Strategy
and Research, Global Medical Affairs, Allergan
| Corporate Overview 4
Board of Directors
Cameron Durrant, MD, MBAChairman
| Corporate Overview 5
Ronald Barliant, JDo Of Counsel at Goldberg Kohn, extensive experience representing debtors and
creditors in complex bankruptcy caseso United States bankruptcy judge for the Northern District of Illinois 1988 to 2002
Dale Chappell, MD, MBAo Managing member of Black Horse Capital Management, LLCo Previously associate at Chilton Investment Company specializing in healthcare
Timothy Morris, CPAo CFO AcelRx Pharmaceuticals; also Head of Business Developmento CFO, SVP Finance and Global Corporate Development VIVUS, Inc.
Ezra Friedbergo Founder and general partner of Multiplier Capital, LPo >20 years investing experience across public and private companies
Two-Pronged Value Model: a Unique Approach To Near-Term Return Propelling Long-Term Value Creation
| Corporate Overview 6
Smart Investment in underappreciated assets
LeverageFDA incentives for better efficiency
Value Options From multiple FDA catalysts like PRV
Value Creation Potential robust commercial return
HuntLate-stage, neglected, rare/orphan assets
Priority Review Vouchers (PRVs) Create Options For Significant Potential Return
| Corporate Overview 7
Highest disclosed sale price so far for PRV is $350MM
Rare Pediatric Diseases (RPD)
Neglected Tropical
Diseases (NTD)
o Holder of PRV can receive priority review for any NDA/BLA
o PRV can be sold to company seeking a competitive jump
o KaloBios open to novel potential PRV transaction structures
| Corporate Overview 8
Funding dead-end, winding down Oct 2015
Martin Shkreli investment late Nov 2015
Shkreli arrest, indictment Dec 2015
Chapter 11 filing Dec 2015
Delisted by Nasdaq Jan 2016 Two new board members Jan 2016
New CEO Mar 2016
Announced Responsible Pricing Model April 2016
Exit from Chapter 11 bankruptcy June 30, 2016
Lenzilumab Phase 1 start July 2016
Shkreli governance agreement July 2016
Shkreli stake extinguished Aug 2016
Current with SEC filings Sept 2016
San Francisco R&D company, mAb platform
FDA meeting Dec 2016 and positive minutes Jan 2017
KaloBios Turnaround Momentum Builds: Rising from the Ashes
Current Pipeline
| Corporate Overview 9
Pre-clin Phase I Phase II Phase III NDA/BLA
Benznidazole (potential PRV)Chagas diseaseAnti-infective
LenzilumabCMML Anti-GM-CSF mAb
Lenzilumab (potential PRV)JMML (peds) – dependent on interim data in CMMLAnti-GM-CSF mAb
Ifabotuzumab (potential PRV)Solid (peds) tumorsAnti-EphA3 mAb
Partnering potential
Intend to seek approval pursuant to 505(b)(2) pathway
Benznidazole
a potential treatment for Chagas disease
| Corporate Overview 10
o On FDA list of NTDs eligible for PRV
o Neglected tropical disease presenting in the US and other nations
o Currently no FDA-approved treatments
o Immediate need for reliable benznidazole supply in US and abroad
CHAGAS disease
| Corporate Overview 11
Insect carries the parasite called Trypanosoma cruzi
Infected individuals in US*
Can also affect swallowing, digestion and cause neurological complications
300,000
* Source: CDC
A chronic, potentially life-threatening infectious disease spread by contact with feces of an infected triatomine insect, called “kissing bugs”
KEY TAKEAWAYS
Significant portion of chronic patients progress to serious heart illness
Benznidazole: the Preferred Treatment Globally for Chagas
| Corporate Overview 12
•Used in tens of thousands of patients
40 years of clinical experience, primarily in Latin America
•Believed to work by inducing free radicals damaging DNA within parasite •Better tolerated in children; side effect profile extremely well known
Efficacy ~60% or more in children and can be higher in young children, according to published studies
Only available in US via special protocol with CDC
Drug supply believed to be sporadic, product difficult to obtain
N
NHN
N
Benznidazole Strategy, Development Plan On Track
| Corporate Overview 13
Positive FDA Guidance 505(b)(2) Pathway PRV Eligibility Confirmed Progress to Submission
☛ Received minutes from productive meeting
☛ Acceptable to FDA
☛ Expect no clinical efficacy or safety studies
☛ Currently expected to be eligible if approved for Chagas disease
☛ Expect to progress expeditiously toward a submission
2016 2017H1 H2 H1 H2
Manufacturing (CMC)Analytical methods developmentDrug substanceDrug product Stability studies
Non-Clinical (Non-Human) Metabolism / PKToxicity assessmentT.cruzi biologyEnvironmental assessment
ClinicalSummary of relevant clinical studiesRight of Reference & data extraction for studiesHuman bioavailability (BA) Study
RegulatoryPre-IND meeting & open INDOrphan drug status applicationPediatric planPre-NDA meetingAssembly of NDA & NDA filing
Benznidazole Target Approval Plan
| Corporate Overview 14
Pre-IND meeting IND submission
NDA filing 2018
Lenzilumab
a potential pipeline in a product
| Corporate Overview 15
o High unmet need
o Patients typically unsuitable for stem cell transplant
o 40-90% patients show hypersensitivity to GM-CSF
CMML overview
| Corporate Overview 16
Recently classified as separate disease with distinct natural history*
Median overall survival rate in months
Newly diagnosed US patients per year
Age at diagnosis
~1,100
* Formerly classified as subtype of the myelodysplastic syndromes (MDS)
60
a rare hematologic cancer
20
KEY TAKEAWAYS
o No FDA-approved treatment
o Clear unmet need
o Potential for a rare pediatric disease PRV
o GM-CSF hypersensitivity is hallmark
o Pediatric oncology largely ignored in clinical development
JMML overview
| Corporate Overview 17
Event-free survival rate at 5 years (with bone marrow transplant)
New US cases per year
Age of majority of patients at diagnosis is 4 years or younger
~420
very rare, frequently lethal pediatric leukemia
~52%
KEY TAKEAWAYS
Lenzilumab is a Potent Inhibitor of GM-CSF Function In Vitro
| Corporate Overview 18
Inhibits GM-CSF-dependent Proliferation of Human TF-1 Leukemia Cells
Blocks GM-CSF-induced STAT5 Phosphorylation in Human TF-1 Leukemia Cells
Source: KaloBios laboratory notebook
STAT5 phosphorylation detection in TF-1 Cells by Flow Cytometry
Inhibition of STAT5 phosphorylation by lenzilumab in TF-1 Cells (+ 2 ng/mL GM-CSF)
0.001 0.01 0.1 1 10Lenzilumab (µg/mL)
25
20
15
10
0
5
40
30
20
10
0
MFI
0.01 0.1 1 10 100GM-CSF (ng/mL)
Lenzilumab
0.1 1 10 100 1000lgG (pM)
100
50
0
Lenzilumab
Control antibody
% G
row
th
Source: KaloBios pre-clinical study
MFI = mean fluorescence intensity
Lenzilumab: a Pipeline in a Drug Opportunity
| Corporate Overview 19
JMML
ALPS / RALD
Cytokine Release Syndrome
AMML
RASopathies(e.g. CFC syndrome, Costello syndrome)
Noonan Syndrome with PTPN11
CMML
CML
Plexiform Neurofibroma
Pediatric Multiple Sclerosis
US ADDRESSABLE POPULATION
325350
500
600
700
8001,900
5,800
24,000
40,000
Ifabotuzumab
significant potential for partnering and development
| Corporate Overview 20
Ifabotuzumab Has Four Postulated Mechanisms of Action
| Corporate Overview 21
ADCC Mediated Killing of Tumor Cells
Direct Apoptosis of Tumor Cells
Disruption of Tumor Vasculature through Cell
Rounding
Anti-fibrotic EffectKB004 has 4 Postulated Mechanisms of Action (MOA)
1. ADCC Mediated Killing of Tumor Cells
2. Direct Apoptosis ofTumor Cells
NK Cell
ADCC
KB004
EphA3
KB004
EphA3
KB004
EphA3
3. Disruption of Tumor Vasculature through
Cell Rounding
4. Anti-fibrotic Effect
KB004
EphA3
Ifabotuzumab
KB004 has 4 Postulated Mechanisms of Action (MOA)
1. ADCC Mediated Killing of Tumor Cells
2. Direct Apoptosis ofTumor Cells
NK Cell
ADCC
KB004
EphA3
KB004
EphA3
KB004
EphA3
3. Disruption of Tumor Vasculature through
Cell Rounding
4. Anti-fibrotic Effect
KB004
EphA3
Ifabotuzumab
KB004 has 4 Postulated Mechanisms of Action (MOA)
1. ADCC Mediated Killing of Tumor Cells
2. Direct Apoptosis ofTumor Cells
NK Cell
ADCC
KB004
EphA3
KB004
EphA3
KB004
EphA3
3. Disruption of Tumor Vasculature through
Cell Rounding
4. Anti-fibrotic Effect
KB004
EphA3
Ifabotuzumab
KB004 has 4 Postulated Mechanisms of Action (MOA)
1. ADCC Mediated Killing of Tumor Cells
2. Direct Apoptosis ofTumor Cells
NK Cell
ADCC
KB004
EphA3
KB004
EphA3
KB004
EphA3
3. Disruption of Tumor Vasculature through
Cell Rounding
4. Anti-fibrotic Effect
KB004
EphA3
Ifabotuzumab
Humaneered® monoclonal antibody for Ephrin type-A receptor 3 (EphA3)
Capitalization Summary as of 1/9/17
| Corporate Overview 22
Debt $4.3 million
Common Shares Outstanding 14.9 million
Stock Options (WAEP $5.82) 2.1 million
Warrants (WAEP $14.24) 0.3 million
Fully Diluted Shares Outstanding 17.3 million
Note: share and per share amounts as of 9/30/16 – last 10-Q filing Debt includes term loan financing closed on 12/21/16
Demonstrated History of Swift Execution in Challenging Circumstances
| Corporate Overview 23
JUN JUL AUG SEP DEC
Emerge in six months from complex Chapter 11 bankruptcy
Lenzilumab Phase 1 trial started
Extinguished Martin Shkreli’s stake in the company
Current with Securities and Exchange Commission periodic reporting requirements
Assembled and filed three 10-Qs and a 10-K within three months
Productive FDA meeting on benznidazole
30 24 29 23 6
2 0 1 6
Summary
Unique near-term/long-term potential value creation opportunity
Thoughtful business strategy that leverages existing U.S. regulatory and development incentives
Attractive asset portfolio with multiple potential milestones and partnering prospects
Deeply committed and focused new management team with an unwavering commitment to success
| Corporate Overview 24